Rollover Study for Continuing NBI-98854 Administration in Pediatric Subjects With Tourette Syndrome

Open-Label Rollover Study for Continuing NBI-98854 Administration for the Treatment of Pediatric Subjects With Tourette Syndrome

This is an open-label, rollover study to collect long-term safety, tolerability, and investigator- and participant-reported pharmacodynamic (PD) data after chronic administration of NBI-98854 in pediatric participants with Tourette Syndrome (TS), as well as to provide open-label access to NBI-98854 for the treatment of TS for pediatric participants who have taken part in a Phase 2 NBI-98854 study.

Study Overview

• Status: Terminated
• Conditions: Tourette Syndrome
• Intervention / Treatment: Drug: NBI-98854

• Study Type: Interventional
• Enrollment (Actual): 6
• Phase: Phase 2

Contacts and Locations

Study Locations

• Puerto Rico
  • San Juan, Puerto Rico, 00926
    • Neurocrine Clinical Site
• United States
  • California
    • Anaheim, California, United States, 92805
      • Neurocrine Clinical Site
    • San Diego, California, United States, 92108
      • Neurocrine Clinical Site
  • Florida
    • Gulf Breeze, Florida, United States, 32561
      • Neurocrine Clinical Site
    • Hialeah, Florida, United States, 33013
      • Neurocrine Clinical Site
  • Illinois
    • Chicago, Illinois, United States, 60634
      • Neurocrine Clinical Site
    • Naperville, Illinois, United States, 60563
      • Neurocrine Clinical Site
  • Nebraska
    • Lincoln, Nebraska, United States, 68526
      • Neurocrine Clinical Site
  • Texas
    • Dallas, Texas, United States, 75243
      • Neurocrine Clinical Site
    • Houston, Texas, United States, 77058
      • Neurocrine Clinical Site

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 6 years to 18 years (Child, Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Have a clinical diagnosis of Tourette Syndrome (TS)
2. Have participated in the NBI-98854-TS2004 or NBI 98854-TS2005 Phase 2 studies
3. If using maintenance medication(s) for TS and/or TS spectrum diagnoses (e.g. obsessive-compulsive disorder [OCD], Attention-Deficit Hyperactivity Disorder [ADHD]), be on stable doses
4. Be in good general health
5. Subjects of childbearing potential who do not practice total abstinence must agree to use hormonal or two forms of nonhormonal contraception (dual contraception) consistently during the screening, treatment and follow-up periods of the study

Exclusion Criteria:

1. Have an active, clinically significant unstable medical condition within 1 month before screening
2. Have a known history of long QT syndrome or cardiac arrhythmia
3. Have a known history of neuroleptic malignant syndrome
4. Have a cancer diagnosis within 3 years prior to screening (some exceptions allowed)
5. Have an allergy, hypersensitivity, or intolerance to VMAT2 inhibitors
6. Have a blood loss ≥250 mL or donated blood within 56 days, or donated plasma within 7 days, before the start of the study
7. Have a known history of substance dependence, substance (drug) or alcohol abuse within 3 months before the start of the study
8. Have a significant risk of suicidal or violent behavior
9. Have received an investigational drug within 30 days before the start of the study or plan to use an investigational drug (other than NBI-98854) during the study
10. Are currently participating in another NBI-98854 clinical study
11. Are pregnant (for females)

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: NBI-98854; NBI-98854 administered once daily for up to 96 weeks	Drug: NBI-98854; vesicular monoamine transporter 2 (VMAT2) inhibitor; Other Names: Valbenazine

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Number of Participants With Treatment-Emergent Adverse Events (TEAEs)	A TEAE is an adverse event not present prior to the initiation of study drug dosing, or is an already present event that worsens either in intensity or frequency following the initiation of study drug dosing.	Up to 16 Weeks

Collaborators and Investigators

• Sponsor: Neurocrine Biosciences

Study record dates

Study Major Dates

• Study Start (Actual): October 17, 2018
• Primary Completion (Actual): February 18, 2019
• Study Completion (Actual): February 18, 2019

Study Registration Dates

• First Submitted: November 5, 2018
• First Submitted That Met QC Criteria: November 5, 2018
• First Posted (Actual): November 6, 2018

Study Record Updates

• Last Update Posted (Actual): April 19, 2022
• Last Update Submitted That Met QC Criteria: March 25, 2022
• Last Verified: March 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Mental Disorders; Pathologic Processes; Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Disease; Genetic Diseases, Inborn; Basal Ganglia Diseases; Movement Disorders; Neurodegenerative Diseases; Heredodegenerative Disorders, Nervous System; Neurodevelopmental Disorders; Tic Disorders; Syndrome; Tourette Syndrome
• Other Study ID Numbers: NBI-98854-TS2007

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No